Amid rising complaints that drug makers are exploiting loopholes to win approval of so-called orphan drugs, the Food and Drug Administration plans to close at least one of them.
In a blog post, FDA Commissioner Scott Gottlieb acknowledged concerns that the Orphan Drug Act, which allows the agency to approve medicines for treating rare diseases affecting fewer than 200,000 people, needs fixing. As he put it, some drug makers are using orphan designations “as a way to sidestep other important public health goals set out by Congress.”
One criticism, for instance, is that some drugs may have been submitted for FDA approval with the ultimate intention of being used more broadly than if they were designed only for rare disease populations. And Kaiser Health News found that more than 70 were drugs first approved by the FDA for mass market use and were later approved as orphans.
This article is exclusive to STAT+ subscribers
Unlock this article — plus in-depth analysis, newsletters, premium events, and networking platform access.
Already have an account? Log in
Already have an account? Log in
About the Author Reprints
To submit a correction request, please visit our Contact Us page.
STAT encourages you to share your voice. We welcome your commentary, criticism, and expertise on our subscriber-only platform, STAT+ Connect